Transthyretin (TTR)-Mediated AmyloidosisPhase 1NCT02053454
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
A Phase 1 study testing patisiran (ALN-TTR02) and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin (TTR)-Mediated Amyloidosis, run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
Trial summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects Read more about this study
- Who is it for?
- Ages 20 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Transthyretin (TTR)-Mediated Amyloidosis
- What is being tested?
- patisiran (ALN-TTR02) and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jan 2014to Jun 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Healthy Japanese adult males and females aged 20 to 65 years, inclusive (The subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan);
- Subjects who are healthy as determined by clinical assessments;
- Females subjects must be of non-childbearing potential;
- Males with partners of child-bearing potential, must agree to use appropriate contraception.
Who cannot join
- Subjects with a history of serious mental illness;
- Subjects who have a clinically relevant medical or surgical history;
- Subjects with a positive screen for alcohol or drugs of abuse;
- Subjects with safety laboratory test results deemed clinically significant;
- Subjects with known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection;
- Subjects who have received an investigational agent within the 3 months prior to study entry.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis
- Being tested
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Keywords
- RNAi therapeutic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
patisiran (ALN-TTR02)Drug
Ascending doses administered by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)Drug
Calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| patisiran (ALN-TTR02) | Active comparator | Not described Receives: Drug: patisiran (ALN-TTR02) |
| Sterile Normal Saline (0.9% NaCl) | Active comparator | Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Measured: Up to 28 days
Secondary outcomes (8)
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Measured: Up to 90 days
Observed maximum concentration (Cmax) of ALN-TTR02
Measured: Up to 90 days
Time of observed maximum concentration (tmax) of ALN-TTR02
Measured: Up to 90 days
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Measured: Up to 90 days
Terminal elimination half-life (t1/2) of ALN-TTR02
Measured: Up to 90 days
Systemic clearance (CL) of ALN-TTR02
Measured: Up to 90 days
Volume of distribution (V) of ALN-TTR02
Measured: Up to 90 days
Renal clearance (CLR) of ALN-TTR02
Measured: Up to 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2014 (estimated)
- Study startJan 2014
- Primary completionMar 2014
- Study completionJun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin (TTR)-Mediated AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jared Gollob, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02053454
- Sponsor's protocol ID
- ALN-TTR02-005
- Status last verified
- May 2015
- First submitted
- 24 Jan 2014
- Last updated
- 25 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers
- ClinicalTrials.gov ID
- NCT02053454
- Acronym / sponsor ID
- ALN-TTR02-005
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis
- Keywords
- RNAi therapeutic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2014
- Primary completion
- Mar 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 3 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| patisiran (ALN-TTR02) | Active comparator | Drug: patisiran (ALN-TTR02) |
| Sterile Normal Saline (0.9% NaCl) | Active comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 20 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Healthy Japanese adult males and females aged 20 to 65 years, inclusive (The subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan);
- Subjects who are healthy as determined by clinical assessments;
- Females subjects must be of non-childbearing potential;
- Males with partners of child-bearing potential, must agree to use appropriate contraception.
Key exclusion criteria (6 listed)
- Subjects with a history of serious mental illness;
- Subjects who have a clinically relevant medical or surgical history;
- Subjects with a positive screen for alcohol or drugs of abuse;
- Subjects with safety laboratory test results deemed clinically significant;
- Subjects with known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| patisiran (ALN-TTR02)Ascending doses administered by intravenous (IV) infusion | Drug | patisiran (ALN-TTR02) |
| Sterile Normal Saline (0.9% NaCl)Calculated volume to match active comparator | Drug | Sterile Normal Saline (0.9% NaCl) |
Primary outcome
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Time frame: Up to 28 days
8 secondary outcomes
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Time frame: Up to 90 days
Observed maximum concentration (Cmax) of ALN-TTR02
Time frame: Up to 90 days
Time of observed maximum concentration (tmax) of ALN-TTR02
Time frame: Up to 90 days
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Time frame: Up to 90 days
Terminal elimination half-life (t1/2) of ALN-TTR02
Time frame: Up to 90 days
Systemic clearance (CL) of ALN-TTR02
Time frame: Up to 90 days
Volume of distribution (V) of ALN-TTR02
Time frame: Up to 90 days
Renal clearance (CLR) of ALN-TTR02
Time frame: Up to 90 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jared Gollob, MD study director
Alnylam Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing patisiran (ALN-TTR02) and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin (TTR)-Mediated Amyloidosis, run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation (time frame: Up to 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2015.